Tech Session VI: Nanomedicine and Nanoscale Delivery (Focus: Gene)
The modular platform to deliver bio-orthogonal macromolecular conjugates (BMCs) for therapeutic gene editing
Thursday, July 17, 2025
6:19 PM - 6:30 PM EDT
Location: 120 B/C
Introduction: Genome editing in human hematopoietic stem and progenitor cells (HSPCs) holds the potential to elucidate gene-function relationships through precise genetic manipulation and offers a promising avenue for curing currently intractable hematological disorders. The CRISPR-Cas system has revolutionized genome engineering; however, its clinical application has been limited by challenges in delivery and target specificity. These obstacles may be overcome through biochemical engineering of the CRISPR-Cas system, incorporating cell-penetrating peptide (CPP) domains and specificity ligands.
Learning Objectives:
At the completion of this activity, participants will know
learn about synthetic strategies in assembling heterotri-functional Cas9 tripartite constructs.
get inspired on principles of ribonucleoprotein delivery for gene editing.
Danmeng Luo – Senior Research Scientist, Memorial Sloan Kettering Cancer Center; Hannah Major-Monfried – Pediatric Hematology-Oncology Fellow, Memorial Sloan Kettering Cancer Center; Sophia Rha – Research Technician, Memorial Sloan Kettering Cancer Center; Karthikeyan Ponnienselvan – PhD, University of Massachusetts Medical School; Stacy Maitland – Research Associate, University of Massachusetts Medical School; Jack Ralls – PhD candidate, Weill Cornell Medical College; Daniel Bauer – Associate Professor, Boston Children’s Hospital; Scot Wolfe – Professor, University of Massachusetts Chan Medical School; Alex Kentsis – Director, 4Tow Center for Developmental Oncology, Memorial Sloan Kettering Cancer Center
